Transforming health through precision RNA editing
We are working to realize RNA editing’s full potential to improve human health.
Advances in genomics have identified harmful genetic variants that increase a person’s chances of developing disease, as well as beneficial variants that drive healthier outcomes, like preventing elevated cholesterol.
AIRNA’s RNA editing approach has unique potential to develop medicines that either repair harmful variants, or introduce beneficial variants to improve health across broad populations. Our therapeutics are designed to be administered through convenient and safe dosing, and to be withdrawn if desired.
Rare Genetic Disorders
Common Conditions
A PRECISION APPROACH FOR AATD
Our therapeutics are designed to deliver best-in-class solutions for patients.
Alpha-1 antitrypsin deficiency (AATD) is an inherited disease caused by insufficient levels of functional alpha-1 antitrypsin protein, or AAT, due to a rare, harmful genetic variant. This deficiency can lead to severe lung and liver diseases, both of which lack sufficient treatment options.
AATD is particularly suited to RNA editing. Our lead program aims to provide a functional cure for patients by repairing the disease-driving genetic variant underlying the condition. Our therapeutic is designed to precisely reverse this change and restore functional AAT protein production.
Pipeline
Our broad pipeline aims to repair disease-driving genetic variants or enable superior health by introducing beneficial forms of RNA.
Learn how small changes to RNA could make a big impact on disease treatment